Results from a Phase 2 clinical trial investigating EPI-743, a therapy developed by BioElectron, have shown that its long term usage may improve neurological function and prevent disease progression relative to the natural course of Friedreich’s ataxia (FA).
EPI-743 is a potent antioxidant, which can regulate the response of key enzymes involved in the processes of oxidative stress, inflammation and cell death. This randomised, placebo-controlled Phase 2 trial tested the safety and efficacy of the drug on 63 adults with genetically confirmed FA. The therapy was found to be safe and well-tolerated with no reported serious adverse events or toxicity related to treatment.
Results initially failed to reveal any significant differences between those who took EPI-743 or placebo in any of the measured clinical parameters. As a result, post-hoc analysis, including only patients who had shown improvement of at least five points per year on the FARS-Neuro (a threshold indicative of the natural course of disease progression) was performed. This demonstrated that significantly more patients on EPI-743 experienced an improvement of three points or more than placebo, and significantly fewer experienced a three-point decrease.